Over 500,000 people in the United States are afflicted with Parkinson’s disease (PD), a neurodegenerative disorder characterized by the death of (DA) dopaminergic neurons in specific regions of the brain. Patients with PD suffer from a disheartening loss of independence due to symptoms such as tremor, slowed movements, stiffness, and difficulty walking. 1-2% of people over the age of 60 will be diagnosed with PD, and the current medical and surgical treatments provide only temporary, symptomatic relief. Since we have not yet been able to address the underlying loss of DA neurons, the disabling symptoms eventually return. DA neuron transplantation has great promise as a PD therapy, and stem cell research has made it possible to produce sufficient quantities of DA neurons. To successfully use DA neurons for the treatment of PD, the neurosurgical transplantation techniques must be safe, efficient, and carefully studied. We have recently innovated a new device for cell transplantation that utilizes the most sophisticated and modern image-guided methods available. Here, we propose moving this work forward to develop DA neuron dosing parameters and surgical strategies that allow us to “tailor” the transplant to individual patient needs. We will also investigate how to best integrate DA neuron transplantation with other current PD therapies. Our work may also benefit the treatment of a wide range of diseases including Alzheimer’s, Huntington’s, multiple sclerosis, and stroke.

Statement of Benefit to California:

Parkinson's disease (PD) is the second most common neurodegenerative disease, and there is currently no cure. Between 1-2% of California citizens over the age of 60 suffer from this disabling disease. The annual national cost of PD has been estimated to be $10.8 billion, creating a tremendous burden on both the national and California state economies. The citizens of California have invested generously into stem cell research for the treatment of human disorders including Parkinson’s disease. In order to successfully convert laboratory research into human therapies, there are certain “practical” hurdles that must be overcome. Our studies of basic cell transplantation parameters, neurosurgical approaches, and clinical trial designs will be crucial for the future treatment of Parkinson’s disease. Furthermore, our work will develop general clinical-surgical principles and methodologies that may benefit the development of cell therapies for Alzheimer’s disease and other important neurological disorders that afflict Californians.